How One Enterprise Ensures Medical Products for Emergencies

by David R. Howell & Joanna M. PrasherTue, April 28, 2015

Pandemic influenza, an aerosolized anthrax attack, a nuclear detonation, chemical or radiological exposure, and other known and emerging threats and disasters are all potential threats to the United States. To combat these, one enterprise – comprising many collaborating federal agencies – is preparing to provide the necessary medical products when and where they are needed.

As the Ebola outbreak spread to multiple countries in West Africa and became a public health emergency of international concern, federal agencies in the United States pulled together quickly to review the vaccines, diagnostics, and therapeutics the agencies were supporting. Collectively known as medical countermeasures, these products were in various states of early development; none had reached clinical trials. The agency representatives quickly made decisions about how to use the authority, funding, and technical expertise from each agency to move as many products as rapidly as possible into clinical trials and get them into the hands of doctors and patients to prevent or treat this potentially deadly infection.

The National Institutes of Health (NIH), Biomedical Advanced Research and Development Authority (BARDA), Centers for Disease Control and Prevention (CDC), and Department of Defense provided funding, facilities, and technical support to private sector companies to bring three vaccines to phase 1 and 2 clinical trials to test safety and efficacy. BARDA also began working with the companies to scale up and improve manufacturing processes and support other advanced development work to increase the supply of candidate countermeasures. BARDA and NIH provided funding and technical assistance to private companies to move forward with development of therapeutics, including ZMapp, a monoclonal antibody cocktail, the components of which are manufactured in tobacco plants. BARDA also is partnering with companies on ways to make similar therapeutics in Chinese hamster ovary (CHO) cells, a modern, scalable monoclonal antibody production platform that will facilitate manufacturing large quantities of the drug.

An Enterprise, a Strategy & a Plan
That collaboration, speed, and empowerment are the strengths of the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE). In one way or another, all of the agencies involved in the PHEMCE support the development and use of medical countermeasures for military or civilian populations or both. The PHEMCE is led by the U.S. Department of Health and Human Services (HHS) Office of the Assistant Secretary for Preparedness and Response (ASPR), including ASPR’s BARDA, and includes three primary HHS internal agency partners: the CDC, the Food and Drug Administration, and the NIH. Other partners include the Departments of Defense, Veterans Affairs, Homeland Security, and Agriculture.

This group plans together to ensure that development of the most critical medical products takes place as quickly as possible and in a coordinated fashion. PHEMCE processes, which facilitate dialogue between developers and end users, ensure that these products can be effectively used to address health threats from chemical, biological, radiological, or nuclear agents and emerging infectious diseases (including pandemic influenza). Members of the PHEMCE consult with state and local health agencies and other ultimate users of the medical countermeasures to ensure the federal government is pursuing medical countermeasures that make sense in the field. The type and amounts of medical countermeasures to stockpile is determined through a PHEMCE-wide process, led by ASPR, which considers the anticipated need, the national abilities to use the medical countermeasures effectively, and the benefits and limitations of different types of products in a public health emergency. An annually updated plan, known as the PHEMCE Strategy and Implementation Plan, provides the blueprint the federal agencies will follow to make the best use of available resources to enhance national health security.

The PHEMCE Strategy and Implementation Plan not onlyentifies goals and objectives, but also describes the activities and programs PHEMCE agencies will undertake over the next five-year horizon toentify, develop, stockpile, and effectively use the medical countermeasures communities across the nation will need to protect health from manmade or naturally occurring threats. In addition, the PHEMCE Strategy and Implementation Plan describes progress federal agencies are making against previous priorities, including developing products to meet the medical needs of particularly at-risk populations, such as children, pregnant women, and people with weakened immune systems. The plan also outlines what the federal government has purchased and what can be made available to local communities facing a public health emergency.

Product Development & Strategic Stockpiles
The drugs, vaccines, and medical devices people use every day require years, often decades, to develop, and private industry spends billions of dollars bringing these products to market. For each product that reaches the market, many others fail, for any of a variety of technical, business, and regulatory reasons. Developing products needed for public health emergencies can be even more difficult. Yet, by collaborating through PHEMCE, federal agencies have made tremendous progress since the first PHEMCE Strategy and Implementation Plan in 2007. More than 80 products – including new es of drugs – have reached advanced development stages and a dozen types of products have been added to the Strategic National Stockpile in just eight years. These products will help combat anthrax, smallpox, botulism, pandemic influenza, radiological and nuclear incidents, and chemical nerve agents. Twenty products have moved through advanced development with support from PHEMCE agencies like BARDA to receive approval or licensure from the Food and Drug Administration and are either stockpiled in the Strategic National Stockpile or available on the commercial market for nonemergency uses.

To ensure the safest and most effective use of limited medical countermeasures following an attack, PHEMCE partners developed up-to-date clinical guidance for using these medical countermeasures in children, pregnant women, and/or the general population under mass-casualty conditions caused by agents such as anthrax, and bacterial infections such as glanders and meliodosis. In 2015, the agencies are collaborating on clinical guidance for products to be used in mass-casualty incidents involving botulism or to treat blood-related injuries after a radiological or nuclear incident.

To ensure the federal government is pursuing products that can be used effectively in public health emergencies, the PHEMCE agencies are collaborating to strengthen the feedback loop between end users and product developers. BARDA – the PHEMCE agency that supports the last stages of product development – partners with private industry and end-user organizations to ensure that product development plans take into account the most up-to-date utilization policies, response strategies, regulatory guidance for use, and other relevant factors.

ASPR, in collaboration with the Department of Homeland Security’s Federal Emergency Management Agency, is supporting the development of regional medical countermeasure plans to complement dispensing plans developed by ten large metropolitan areas currently included in Tier One of the Urban Areas Security Initiative areas under the Cities Readiness Initiative program. This planning initiative develops community-based operational plans under which the federal government will rapidly augment the capabilities of the affected area in response to an incident such as a widespread aerosolized anthrax attack. Partners involved in this effort include state and local health department, regional healthcare coalitions, emergency management offices, public information offices, and federal agencies.

Such planning may include closed or open points of dispensing. Closed points of dispensing rely on large private or public sector employers in the community to receive medical countermeasures from government stockpiles and distribute those countermeasures to employees and their families. Open points of dispensing are open to the public to pick up medical countermeasures. Plans to dispense medical countermeasures are essential to national health security, ensuring that the medical countermeasures developed or stockpiled by state and federal governments reach impacted community members as quickly as possible after an incident.

International Collaboration
During public health emergencies, such as the 2009 H1N1 pandemic, the international community may request medical countermeasures from the United States. PHEMCE agencies partnered to develop the official U.S. policy for responding to these international requests in order to share public health emergency countermeasures. The policy covers how the U.S. government receives, considers, decides, communicates, and responds to such requests. Vital efforts continue and are reflected in the latest PHEMCE Strategy and Implementation Plan. Learn more about the work completed, underway, or planned – from Ebola to influenza and from sarin to radiation – under the 2014 PHEMCE Strategy and the Implementation Plan.

David R. Howell (pictured), Ph.D., is a senior policy analyst for the Office of the Assistant Secretary for Preparedness and Response, Office of Policy and Planning, Division of Medical Countermeasure Strategy and Requirements. He supports policy and strategy development for the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE). Previously, he served as a counterterrorism analyst at the Department of Homeland Security. Prior to that, he was an analyst at the RAND Corporation. He holds a B.S. in physics from Bucknell University, an M.S. in international affairs from Georgia Tech, and a Ph.D. in policy analysis from the Pardee RAND Graduate School.

Joanna M. Prasher, Ph.D., is the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE) branch chief for the Medical Countermeasures Strategy and Requirements Division within the Office of Policy and Planning, Office of the Assistant Secretary for Preparedness and Response, U.S. Department of Health and Human Services. She supports strategic planning for civilian medical countermeasure requirements, development, stockpiling, and effective use. She received a bachelor’s degree in biology from Kalamazoo College (Magna Cum Laude) and a doctorate in experimental pathology from the University of Utah. She conducted postdoctoral work at Erasmus University in The Netherlands before joining the Department of Health and Human Services in 2004.